CA3141900C - Compositions et procedes de regulation selective de genes - Google Patents

Compositions et procedes de regulation selective de genes Download PDF

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CA3141900C
CA3141900C CA3141900A CA3141900A CA3141900C CA 3141900 C CA3141900 C CA 3141900C CA 3141900 A CA3141900 A CA 3141900A CA 3141900 A CA3141900 A CA 3141900A CA 3141900 C CA3141900 C CA 3141900C
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certain embodiments
scn1a
seq
dbd
expression
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CA3141900A1 (fr
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Stephanie TAGLIATELA
Anne TANENHAUS
Kartik RAMAMOORTHI
Andrew Young
David OBERKOFLER
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Encoded Therapeutics Inc
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Encoded Therapeutics Inc
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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Abstract

La présente invention concerne des facteurs de transcription modifiés pour la régulation à la hausse sélective de SCN1a et leurs utilisations pour le traitement de maladies et de troubles, tels que le syndrome de Dravet. L'invention concerne également des sites de liaison de microARN et leurs utilisations pour une expression sélective dans des neurones à parvalbumine.
CA3141900A 2019-05-29 2020-05-29 Compositions et procedes de regulation selective de genes Active CA3141900C (fr)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962854238P 2019-05-29 2019-05-29
US62/854,238 2019-05-29
US201962857727P 2019-06-05 2019-06-05
US62/857,727 2019-06-05
US202063008569P 2020-04-10 2020-04-10
US63/008,569 2020-04-10
PCT/US2020/035431 WO2020243651A1 (fr) 2019-05-29 2020-05-29 Compositions et procédés de régulation sélective de gènes

Publications (2)

Publication Number Publication Date
CA3141900A1 CA3141900A1 (fr) 2020-12-03
CA3141900C true CA3141900C (fr) 2023-06-06

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Family Applications (1)

Application Number Title Priority Date Filing Date
CA3141900A Active CA3141900C (fr) 2019-05-29 2020-05-29 Compositions et procedes de regulation selective de genes

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US (2) US12516350B2 (fr)
EP (2) EP3976798B1 (fr)
JP (1) JP7432621B2 (fr)
KR (1) KR102808368B1 (fr)
CN (1) CN114174520B (fr)
AU (2) AU2020282352B2 (fr)
BR (1) BR112021024055A2 (fr)
CA (1) CA3141900C (fr)
CL (1) CL2021003151A1 (fr)
CO (1) CO2021017544A2 (fr)
ES (1) ES3062644T3 (fr)
IL (1) IL288313A (fr)
MX (1) MX2021014460A (fr)
PL (1) PL3976798T3 (fr)
SG (1) SG11202113048SA (fr)
TW (1) TWI872078B (fr)
WO (1) WO2020243651A1 (fr)

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JP7401432B2 (ja) 2017-12-01 2023-12-19 エンコーデッド セラピューティクス， インコーポレイテッド 操作されたｄｎａ結合タンパク質
SG11202103102WA (en) 2018-09-26 2021-04-29 California Inst Of Techn Adeno-associated virus compositions for targeted gene therapy
AU2020228028A1 (en) 2019-02-25 2021-09-30 University Of Massachusetts DNA-binding domain transactivators and uses thereof
WO2020210633A1 (fr) * 2019-04-12 2020-10-15 Encoded Therapeutics, Inc. Compositions et méthodes d'administration de produits thérapeutiques
WO2020243651A1 (fr) 2019-05-29 2020-12-03 Encoded Therapeutics, Inc. Compositions et procédés de régulation sélective de gènes
US20240398985A1 (en) * 2021-09-16 2024-12-05 Novartis Ag Novel transcription factors
EP4151736A1 (fr) * 2021-09-21 2023-03-22 SVAR Life Science AB Nouveau système pour la quantification d'anticorps neutralisants anti-aav
CA3244955A1 (fr) * 2021-12-30 2023-07-06 Regel Therapeutics, Inc. Compositions pour la modulation de l’expression de la sous-unité alpha 1 du canal sodique à tension et leurs utilisations
CN114507693A (zh) * 2022-02-09 2022-05-17 中国人民解放军陆军军医大学第一附属医院 重组腺相关病毒表达载体及其应用
KR20250141200A (ko) * 2023-02-01 2025-09-26 알렌 인스티튜트 전압-게이팅 나트륨 채널 기능의 인테인 매개 재구성
KR20260040173A (ko) * 2023-05-26 2026-03-24 레겔 테라퓨틱스, 인코포레이티드 나트륨 전압 개폐 채널 알파 서브유닛 2의 발현을 조정하기 위한 조성물 및 이의 용도
WO2025019685A2 (fr) * 2023-07-18 2025-01-23 Allen Institute Oligonucléotides antisens et constructions d'expression artificielle pour exprimer un arn dans des neurones inhibiteurs
GB202401827D0 (en) * 2024-02-09 2024-03-27 Ucl Business Ltd Therapy for dravet syndrome
WO2026015815A1 (fr) * 2024-07-12 2026-01-15 Design Therapeutics, Inc. Méthodes de traitement de troubles de l'haplo-insuffisance

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